enGene Reports Third Quarter 2025 Financial Results and Provides Business Update

Achieved target enrollment milestone for LEGEND trial pivotal cohort in high-risk BCG unresponsive NMIBC with carcinoma in situ LEGEND’s pivotal cohort data update planned for 4Q 2025 Detalimogene granted Regenerative Medicine Advanced Therapy (RMAT) designation by FDA Biologic License Application (BLA) submission planned for 2H 2026 Cash and marketable securities of $224.9 million expected to provide runway into 2027 BOSTON & MONTREAL – enGene Holdings Inc. (Nasdaq: ENGN, “enGene” or the “Company”), a clinical-stage, non-viral gene therapy company, today announced its financial results for the third quarter ended July 31, 2025, and provided a business update. “Reaching target enrollment in LEGEND’s pivotal Cohort 1 and securing RMAT designation are important milestones that mark our continued momentum,” said Ron Cooper, Chief Executive Officer of enGene. “We look forward to providing a data update from the LEGEND pivotal cohort later this year. These advances bring us closer to our planned BLA filing in 2026 with the ultimate goal of delivering detalimogene to patients as a therapy designed for efficacy, safety, and ease-of-use.” Recent Corporate Updates LEGEND study enrollment update: The Company announced that it achieved its target enrollment milestone of 100 patients with high-risk, BCG-unresponsive NMIBC carcinoma in-situ (CIS) with or without concomitant papillary disease for the pivotal cohort of its ongoing, open-label, multi-cohort Phase 2 LEGEND trial of detalimogene. Patients in the screening process remain eligible for potential enrollment in Cohort 1. Detalimogene granted RMAT designation: The Company announced that the U.S. Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to detalimogene voraplasmid for the treatment of high-risk (HR), Bacillus Calmette-Guérin (BCG)-unresponsive, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) based on previously disclosed data from the ongoing LEGEND trial. This designation provides enGene with several regulatory advantages, including early and frequent engagement with the FDA and the potential for rolling submission and priority review. Detalimogene was also granted Fast Track designation from the FDA in November 2020. Key board and management appointments: The Company announced the appointment of Philip Astley-Sparke, William Grossman, M.D., Ph.D., and Michael Heffernan R.Ph., to its Board of Directors, and the promotion of Matthew Boyd to Chief Regulatory Officer, Jill Buck to Chief Development Officer, and Katherine Chan, M.D., M.P.H. to Executive Director, Urology Clinical Lead. Their combined experience across gene therapy, oncology, urology, clinical development, and global product launches will support enGene’s strategic transition toward its planned commercialization of detalimogene. Anticipated Milestones Updated preliminary data from LEGEND trial’s pivotal cohort in 4Q 2025. Trial in progress updates from LEGEND’s additional cohorts, including HR-NMIBC patients with CIS who are naïve to treatment with BCG (Cohort 2a); HR-NMIBC patients with CIS who have been exposed to BCG but have not received adequate BCG treatment (Cohort 2b); and BCG-unresponsive HR-NMIBC patients with papillary-only disease (Cohort 3), in 4Q 2025. Planned BLA filing for LEGEND’s pivotal cohort in 2H 2026.

The release as fetched from its publisher. engene.com ↗